Article
Inhibition of the QPCT-PDIA4 axis rescues ΔF508 and N1303K CFTR in cystic fibrosis.
Nature communications - 9 Jun 2026
Sun Le, Rodriguez Larry, Pankow Sandra, Diedrich Jolene, Qin Ke, Zhang Jianan, Wu Xu, Wu Peng, Yates John R
Abstract excerpt
Cystic fibrosis (CF) is a genetic disorder caused by CFTR mutations, most commonly ΔF508, leading to defective ion transport and multisystem pathology. Small-molecule modulators partially restore mutant CFTR function, but therapeutic efficacy remains limited, particularly for N1303K mutation refractory to current treatments. Here, we show that inhibition of the glutaminyl-peptide cyclotransferase (QPCT)-dependent...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.